NeuroVia has commenced a Phase l/ll clinical trial of NV1205 for the treatment of patients with childhood cerebral adrenoleukodystrophy (CCALD).
The trial aims to evaluate the safety, tolerability and pharmacokinetics of increasing doses of NV1205 in patients diagnosed with CCALD.
The trial is to be conducted in eight different countries, including Argentina, Australia, Chile, Colombia, France, the Russian Federation, Ukraine and the UK, and estimated to enrol 25 subjects.
It will include a 30-day screening period starting from the administration of the first dose.
It will also feature a main treatment period of 12 weeks, where subjects will have an initial four-week treatment period at the assigned dose and, if no safety concerns are noted, subjects will continue for another eight weeks of extended safety assessment.
A long-term treatment (LTT) period is also included in the trial to allow the subjects to continue to receive treatment.
The primary endpoint of the trial is the number of participants with treatment-related adverse events as assessed by common terminology criteria for adverse events v4.0 (CTCAE v4.0) over a period of two years.
The trial’s secondary endpoints include area under the curve concentration of NV1205 in plasma over a 12-week period, among others.
It will further evaluate percentage change from baseline in brain lesions assessed as Loes score.
NeuroVia chief development officer John Henderson said: “The study initiation in Australia, Chile and the UK is just the first step in our global clinical development strategy.
“We believe NV1205 has real potential to better serve the X-ALD community as a novel, pharmaceutical treatment for all X-ALD patients.”
NV1205 is an investigational synthetic small molecule designed to restore metabolic activity within the cells by allowing them to process the detrimental, accumulating very long chain fatty acids (VLCFA) and avoid the resulting damage to neuronal and mitochondrial membranes altogether.